Expand all sections

Omaveloxolone

Extended indication

Treatment of Friedreich's Ataxia
Therapeutic value

Possible added value
Registration phase

Registration application pending

Product

Active substance

Omaveloxolone
Domain

Neurological disorders
Reason of inclusion

New medicine (specialité)
Main indication

Muscular diseases other
Extended indication

Treatment of Friedreich's Ataxia
Manufacturer

Reata
Route of administration

Oral
Therapeutical formulation

Capsule
Budgetting framework

Extramural (GVS)
Additional remarks 
Nrf2 activator

Registration

Registration route

Centralised (EMA)
Type of trajectory

Normal trajectory
ATMP

No
Submission date

December 2022
Expected Registration

February 2024
Orphan drug

Yes
Registration phase

Registration application pending

Therapeutic value

Current treatment options

Revalidatie, geen echte behandeloptie.
Therapeutic value

Possible added value
Substantiation

Er is op dit moment nog geen directe behandeling mogelijk. De fase 2 studie is gepubliceerd in 2021 (1). Omaveloxolone verbeterde significant de neurologische functies vergeleken met placebo en was veilig. Recent is er ook een extensie-studie gepubliceerd. Deze resultaten bevestigen de positieve resultaten uit de MOXIe fase 2 en laten een voordeel zien van omaveloxolone behandeling gemeten in mFARS.
Duration of treatment

continuous
Frequency of administration

1 times a day
Dosage per administration

2,5-300 mg
References 
NCT02255435; Ann Neurol. 2021 Feb;89(2):212-225 (1); Mov Disord. 
2023 Feb;38(2):313-320
Efficacy of Omaveloxolone in Friedreich's Ataxia;

Expected patient volume per year

Patient volume

< 340

Market share is generally not included unless otherwise stated.
References 
Spierziekten.nl (1)
Additional remarks 
Friedreich's ataxia komt voor bij 1 op de 50.000 mensen (1). Voor Nederland zou dit dan gaan om 340 mogelijke patiënten.

Expected cost per patient per year

References 
Drugs.com
Additional remarks 
De prijs in de Verenigde Staten is $32,477 voor 90 capsules. De prijs in de Verenigde Staten ligt vaak hoger dan de Nederlandse prijs.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension

Yes
Indication extensions

Mitochondrial disorders; Ocular inflammation; Ocular pain; Malignant melanoma
References 
Adis insight
Additional remarks 
Huidige studies bevinden zich in fase 2.

Other information

There is currently no futher information available.